How Does Evidence-Based Research Influence the Design of Your Studies?
Last week, I participated in a late phase conference where evidence-based medicine was discussed at length. There is definitely an increasing momentum in this area as demonstrated by the recent creation of major initiatives, such as Comparative Effectiveness Research (CER) and the Patient-Centered Outcomes Research Institute (PCORI).
CER is defined as the conduct and synthesis of evidence comparing the benefits and harms of alternative methods to prevent, diagnose, treat and monitor health conditions in real-world settings. The information collected through evidence-based studies is used by regulators to approve or disapprove products; by health agencies to issue clinical guidelines; and by physicians and patients alike to make decisions about clinical care. In addition, sponsors are increasingly using this information to demonstrate the value of new medicines or new medical devices to insurers and payers, as compared to standard clinical care.
PCORI is an independent, non-profit organization authorized by the US Congress to continually seek input from patients and a broad range of stakeholders to guide its work. Patient-centered outcomes research combined with CER promises to enhance decision makers’ ability to fully understand and weigh health care alternatives.
In the context of evidence-based research, both randomized clinical trials (RCTs) and post-approval observational studies can be used to collect data in order to make informed decisions about health policy and clinical care.
RCTs are most appropriate when you need to have a high level of control and certainty. Observational studies and registries are ideal to evaluate the applicability of evidence obtained through RCTs. They also can provide a better understanding of current treatment practices and information through long-term follow-up.
However, they pose unique challenges characterized by a much broader range of patients and sites, significantly more sporadic patient visit schedules and assessments of not only safety and efficacy but also effectiveness and product utilization in a real-world environment.
This situation creates a different paradigm from RCTs and therefore requires a different approach. It is the difference between driving the prototype car on the test track under well-controlled conditions or the production model on busy highways or city streets.
This is the right time to design and implement studies with a clear focus on collecting data directly from patients or their caregivers. But be aware that you may have to take a different route if you are stuck in traffic or if you feel that there is a more scenic road ahead!
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